Myeloma Institute Clinical Trials

At the core of the successful outcomes at the Myeloma Institute is a treatment approach that combines chemotherapy with autologous stem cell transplantation followed by a maintenance strategy. This approach, known as “Total Therapy,” incorporates proven myeloma agents up front to reduce tumor burden during initial treatment to prevent later relapse.

The Myeloma Institute was the first center to achieve truly curative outcomes in multiple myeloma through the novel Total Therapy approach, which encompasses a series of clinical trials designed by and conducted only at the Myeloma Institute. Total Therapy has evolved over the years to include new agents with novel mechanisms of action, yielding significant improvements in overall survival and progression-free survival.

Researchers and physicians at the Myeloma Institute never cease in their commitment in developing new ways to treat multiple myeloma and other related diseases. Our Investigators continue to design clinical trials to answer important scientific questions and to find better ways to treat patients and improve outcomes.

The Myeloma Institute also participates in many collaborative, multi-center clinical trials sponsored by pharmaceutical companies in order to give patients access to some of the latest pharmaceutical advances and to improve outcomes and quality of life.

What is a Clinical Trial?

A Clinical trial (or clinical research) is a research study in which people participate as volunteers to help doctors find ways to improve the health of patients so that they can live longer, healthier lives.

Cancer clinical trials test many types of therapies, such as new drugs or new combinations of treatments. The goal of each study is to answer scientific questions to find better ways to prevent, diagnose, or treat patients with cancer and those who will develop cancer in the future.

All of today’s standard cancer treatments were first shown to be effective in clinical trials. New and better treatments for cancers, such as multiple myeloma, will be discovered only through the continued support of patients who participate in clinical trials.

Clinical trials are sponsored or funded by a variety of organizations or individuals such as physicians, medical institutions, foundations, voluntary groups, and pharmaceutical companies, in addition to federal agencies such as the National Institutes of Health (NIH), the Department of Defense (DOD), and the Department of Veterans Affairs (VA). Trials can take place in a variety of locations, such as hospitals, universities, doctors’ offices, or community clinics.

There are two types of clinical studies.

Observational study: This is a type of study in which participants identified as belonging to study groups are assessed for biomedical or health outcomes. Participants may receive diagnostic, therapeutic, or other types of interventions, but the investigator does not assign participants to specific interventions.

Clinical trial (interventional study): A clinical trial is a study in which participants receive one or more interventions so that researchers can evaluate the effects of the interventions on health-related outcomes. The assignments are determined by the study protocol. Participants may receive diagnostic, therapeutic, or other types of interventions. The FDA (Federal Drug Administration) categorizes clinical trials into phases in order to describe the clinical trial of a drug based on the study’s characteristics, such as the objective and number of participants.

Phase 1 studies emphasize safety. The goal is to find out what the drug’s most frequent and serious adverse events are and, often, how the drug is metabolized and excreted.

Phase 2 studies gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition). For example, participants receiving the drug may be compared with similar participants receiving a different treatment, usually an inactive substance (called a placebo) or a different drug. Safety continues to be evaluated, and short-term adverse events are studied.

Phase 3 studies gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.

Phase 4 studies occur after the FDA has approved a drug for marketing. These include post-market requirement and commitment studies that are required of or agreed to by the sponsor. These studies gather additional information about a drug’s safety, efficacy, or optimal use.

Expanded Access studies are trials in which the FDA allows manufacturers to provide investigational new drugs for patients with serious diseases or conditions who cannot participate in a clinical trial.

Participating in a Clinical Trial

Taking part in a clinical trial is voluntary. Not all clinical trials are right for all patients. A trial may be safe for one patient, but not for another. Each trial has strict rules that the healthcare team must follow in order to determine who is eligible to join the clinical trial. These eligibility criteria include information about a patient’s age, gender, medical test results, current and prior treatments and medicines, treatment duration, length of time since previous treatment, and any other health problems or conditions.

Patients must go through a process called informed consent before being enrolled in a clinical trial. This process involves explanation about important information related to the study, including potential risks and benefits and available alternatives, so that potential participants can make an informed decision about whether or not to enroll. Also emphasized during the process of informed consent is that a clinical study is completely voluntary and that participants can withdraw from a study at any time.

Patients are not required to sign the informed consent form immediately. Patients are encouraged to take time to review the form and to ask questions about anything that might not be clear. Patients can also ask questions at any time during the course of the clinical trial. Similarly, any new risks or side effects that become known during the clinical trial will be explained to participants.